HACKENSACK, N.J., Oct. 13, 2016 /PRNewswire-USNewswire/ --Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), has awarded a $239,000 grant to Dr. Terence Partridge, Professor of Systematic Integrative Biology, Research Center for Genetic Medicine at Children's National Medical Center. This grant will help Dr. Partridge and his team continue their work in understanding exon skipping technology for future trials in Duchenne.

Exon skipping is a promising treatment for Duchenne, with the first FDA accelerated approval in Duchenne recently received by Sarapeta Therapeutics for their therapy Exondys 51. Exon skipping uses antisense agents that specifically skip mutated parts of the gene to prevent disruption of production of the dystrophin protein. Dr. Partridge aims to gain a better understanding of how these agents work in cells to obtain information that will be used to optimize exon skipping therapeutic development.

"PPMD was excited to learn more about the work Dr. Partridge and his team at Children's National are doing. It is important to support work that aims to better understand therapies so that our community can participate in clinical trials that are safe and reflect the latest technology. Given the recent accelerated approval of Exondys 51 and the ongoing potential of exon skipping as a therapy for Duchenne, this work is even more important," said PPMD's Senior Vice President of Research Strategy Abby Bronson.

According to Dr. Partridge, "Exon skipping is the most promising approach that I have come across as a means of treating Duchenne, but has the problem that it is less efficient than we would like it to be. We think that the best way of achieving this is to identify the weak points in the process and attack them individually. This generous funding from PPMD comes at an opportune moment, when we have just developed and validated a good system for conducting such an analysis."

Dr. Partridge and his team will test three different antisense oligonucleotide chemistries in two different versions of the mdx mouse, a milder phenotype and a more severe phenotype which is more representative of Duchenne in humans. Through this series of tests, the team will characterize the dynamics of what happens between administration of the antisense agent and the production and maintenance of dystrophin within the muscle fibers. Specifically, they will look at: efficient delivery of the antisense agent into the muscle myonuclei, the persistence of these agents within the muscle, efficiency of production and longevity of the exon-skipped transcript, and the rate of synthesis of the dystrophin protein and how long it lasts within the muscle. The team hopes to identify any areas where optimization of the therapy could occur, and hopes to make exon skipping more efficient in the future.

Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne.

We invest deeply in treatments for this generation of people affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite and educate the global Duchenne community.

Everything we do—and everything we have done since our founding in 1994—helps people with Duchenne live longer, stronger lives. We will not rest until every person has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne. Follow PPMD on Facebook, Twitter, and YouTube.

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